• What percentage of subjectswill manifest transaminitis and loss of FIX expression at the top dose?
多少百分比的受測者在最高劑量下會顯現轉氨脢升高跟凝血因子表現衰退?
• Can optimization with a gain-of-function mutation reduce the vector dose,thereby reducing immune-mediated transaminitis (FIX Padua variant R338L)?
功能獲得性突變的最佳化能夠降低假體劑量，因而減少抵抗力媒介型(或稱免疫型)轉氨酶炎嗎(凝血因子Padua變體R338L)?
• What percentage of these will respond to prednisolone?
這些受測者有多百分比對抗炎敏有所反應?
• What are the treatment strategies for those with pre-existing antibodies toAAV8? Are there other serotypes available for those with pre-existing immunityand for top-up doses of gene therapy for those with loss of expression withtime? In case of cross reactivity, compartmentalized delivery may yield robustexpression.
對於那些原本就帶有腺病毒載體8型抗體的人有何醫療策略?有沒有那些具既有免疫力的人以及表現隨著時間衰退者基因療法的半生物效應劑量可用的其他血清型呢?若是出現交叉反應，分類傳送可能產生穩健的表現。
Or variants of serotypesobtained by introducing specific mutations in the capsid may reduce crossreactivity.
或者，藉由衣殼內特定抗體的導入所獲得的血清變體可能減少交叉反應。
- 轉氨酶炎（transaminitis，無症狀的血清轉氨酶升高）
- prednisolone (抗炎敏)：一種皮質性類固醇，作用為緩解發炎反應(腫、熱、紅、痛)。
留言列表
